Title :

Long-term prophylaxis in hereditary angioedema management: current practices in France and unmet needs.

Author(s) :

Bouillet, Laurence [Auteur]
Translational Innovation in Medicine and Complexity / Recherche Translationnelle et Innovation en Médecine et Complexité - UMR 5525 [TIMC ]
Centre de référence national des angiœdèmes [CREAK]
Fain, Olivier [Auteur]
CHU Saint-Antoine [AP-HP]
Armengol, Guillaume [Auteur]
CHU Rouen
Aubineau, Magali [Auteur]
Hospices Civils de Lyon [HCL]
Blanchard-Delaunay, Claire [Auteur]
Centre Hospitalier Georges Renon [Niort] [CH Georges Renon Niort]
Dalmas, Marie-Caroline [Auteur]
Centre Hospitalier Universitaire [Strasbourg] [CHU Strasbourg]
De Moreuil, Claire [Auteur]
Hôpital de la Cavale Blanche - CHRU Brest [CHU - BREST ]
Du Thanh, Aurélie [Auteur]
CHU Montpellier = Montpellier University Hospital
Gobert, Delphine [Auteur]
CHU Saint-Antoine [AP-HP]
Guez, Stéphane [Auteur]
Hôpital Pellegrin
Hoarau, Cyrille [Auteur]
Centre Hospitalier Régional Universitaire de Tours [CHRU Tours]
Jaussaud, Roland [Auteur]
Centre Hospitalier Régional Universitaire de Nancy [CHRU Nancy]
Jeandel, Pierre-Yves [Auteur]
Université Côte d'Azur [UniCA]
Maillard, Hervé [Auteur]
Centre Hospitalier Le Mans (CH Le Mans)
Marmion, Nicolas [Auteur]
Centre Hospitalier Universitaire de La Réunion [CHU La Réunion]
Masseau, Agathe [Auteur]
Centre Hospitalier Universitaire de Nantes = Nantes University Hospital [CHU Nantes]
Menetrey, Céline [Auteur]
CH Esquirol [Limoges] [CH Esquirol]
Ollivier, Yann [Auteur]
CHU Caen
Pelletier, Fabien [Auteur]
Centre Hospitalier Régional Universitaire de Besançon [CHRU Besançon]
Plu-Bureau, Geneviève [Auteur]
Hôpital Cochin [AP-HP]
Sailler, Laurent [Auteur]
Centre Hospitalier Universitaire de Toulouse [CHU Toulouse]
Vincent, Denis [Auteur]
Centre Hospitalier Universitaire de Nîmes [CHU Nîmes]
Bouquillon, Benoit [Auteur]
Centre Hospitalier Régional Universitaire [CHU Lille] [CHRU Lille]
Verdier, Edouard [Auteur]
Clerson, Pierre [Auteur]
Soladis
Boccon-Gibod, Isabelle [Auteur]
Centre de référence national des angiœdèmes [CREAK]
Launay, David [Auteur]
Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286

Journal title :

Allergy and Asthma Proceedings

Abbreviated title :

Allergy Asthma Proc

Volume number :

43

Pages :

406-412

Publication date :

2022-07-23

ISSN :

1539-6304

English keyword(s) :

Hereditary angioedema
Administration route
Decision-making
Expert opinion
Long-term prophylaxis
Quality of life
Side effects
Survey
Treatment
Unmet needs

HAL domain(s) :

Sciences du Vivant [q-bio]

English abstract : [en]

Background: Hereditary angioedema (HAE) is characterized by unpredictable and potentially life-threatening attacks of cutaneous and submucosal swelling. Over the past decade, new agents, based on a better understanding of ...
Show more >Background: Hereditary angioedema (HAE) is characterized by unpredictable and potentially life-threatening attacks of cutaneous and submucosal swelling. Over the past decade, new agents, based on a better understanding of the underlying biologic mechanisms of HAE, have changed the face of long-term prophylaxis (LTP). Objective: The objective was to describe current practices and unmet needs with regard to LTP for HAE in expert centers in France. Methods: The study was conducted in France in 2020. Based on their experience with patients with HAE who had visited their center at least once in the past 3 years, physicians from 25 centers who are expert in the management of HAE were requested to fill in a questionnaire that encapsulated their active patient list, criteria for prescribing LTP, and medications used. They were asked about potential unmet needs with currently available therapies. They were asked to express their expectations with regard to the future of HAE management. Results: Analysis was restricted to 20 centers that had an active patient file and agreed to participate. There were 714 patients with C1 inhibitor (C1-INH) deficiency, of whom 423 (59.2%) were treated with LTP. Altered quality of life triggered the decision to start LTP, as did the frequency and severity of attacks. Ongoing LTP included androgens (28.4%), progestins (25.8%), lanadelumab (25.3%), tranexamic acid (14.2%), intravenous C1-INHs (5.6%), and recombinant C1-INH (0.7%). Twenty-nine percent of the patents with LTP were considered to still have unmet needs. Physicians' concerns varied among therapies: poor tolerability for androgens and progestins, a lack of efficacy for tranexamic acid and progestins, dosage form, and high costs for C1-INHs and lanadelumab. Physicians' expectations encompassed more-efficacious and better-tolerated medications, easier treatment administration for the sake of improved quality of life of patients, and less-expensive therapies. Conclusion: Despite the recent enrichment of the therapeutic armamentarium for LTP, physicians still expressed unmet needs with currently available therapies.Show less >

Language :

Anglais

Audience :

Internationale

Popular science :

Non

Administrative institution(s) :

Université de Lille
Inserm
CHU Lille

Collections :

• Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286

Submission date :

2024-01-12T01:00:44Z
2024-03-18T10:36:39Z